Regimens for treating and preventing lysosomal disorders and degenerative disorders of the central nervous system

Abstract

Described herein are dosing regimens and kits for the treatment and/or prevention of lysosomal storage disorders such as Gaucher disease. Also described are dosing regimens and kits for the treatment and/or prevention of degenerative disorders of the central nervous system, such as the synucleinopathies Parkinson's disease or Lewy Body Dementia.

What is claimed is: 
     
       1. A method for treating a patient having low β-glucocerebrosidase (GCase) activity, the method comprising:
 administering to the patient an effective amount of (3R,4R,5S)-5-(difluoromethyl) piperidine-3,4-diol or a salt thereof to enhance the GCase activity in the patient, 
 wherein the effective amount is in the range of about 0.75 mg/kg to about 2.5 mg/kg free base equivalent (FBE) per day and the effective amount is administered less than or equal to 7 days a week. 
 
     
     
       2. The method of  claim 1 , wherein the effective amount is in the range of about 0.75 mg/kg to about 1.5 mg/kg FBE per day. 
     
     
       3. The method of  claim 1 , wherein the (3R,4R,5S)-5-(difluoromethyl) piperidine-3,4-diol or salt thereof is administered 7 days a week. 
     
     
       4. The method of  claim 1 , wherein the (3R,4R,5S)-5-(difluoromethyl) piperidine-3,4-diol or salt thereof is administered orally. 
     
     
       5. The method of  claim 1 , wherein the salt is (3R,4R,5S)-5-(difluoromethyl)piperidine-3,4-diol fumarate. 
     
     
       6. The method of  claim 1 , wherein the (3R,4R,5S)-5-(difluoromethyl) piperidine-3,4-diol or salt thereof is administered about every 24 hours. 
     
     
       7. The method of  claim 1 , wherein the effective amount is about 1 mg/kg FBE per day. 
     
     
       8. The method of  claim 1 , wherein the effective amount is about 0.75 mg/kg to about 1 mg/kg FBE per day. 
     
     
       9. The method of  claim 1 , wherein the patient is diagnosed with Gaucher disease. 
     
     
       10. The method of  claim 1 , wherein the patient is diagnosed with Parkinson's disease. 
     
     
       11. A method for reducing glucosylsphingosine (GlcSph) in a patient having low β-glucocerebrosidase (GCase) activity, the method comprising:
 administering to the patient an effective amount of (3R,4R,5S)-5-(difluoromethyl) piperidine-3,4-diol or a salt thereof, 
 wherein the effective amount is in the range of about 0.75 mg/kg to about 2.5 mg/kg free base equivalent (FBE) per day and the effective amount is administered less than or equal to 7 days a week. 
 
     
     
       12. The method of  claim 11 , wherein the salt is (3R,4R,5S)-5-(difluoromethyl)piperidine-3,4-diol fumarate. 
     
     
       13. The method of  claim 11 , wherein the (3R,4R,5S)-5-(difluoromethyl) piperidine-3,4-diol or salt thereof is administered orally. 
     
     
       14. The method of  claim 11 , wherein the effective amount is about 1 mg/kg FBE per day. 
     
     
       15. The method of  claim 11 , wherein the effective amount is in the range of about 0.75 to about 1.5 mg/kg FBE per day. 
     
     
       16. The method of  claim 11 , wherein the effective amount is about 0.75 mg/kg to about 1 mg/kg FBE per day. 
     
     
       17. The method of  claim 11 , wherein the (3R,4R,5S)-5-(difluoromethyl) piperidine-3,4-diol or salt thereof is administered 7 days a week. 
     
     
       18. The method of  claim 11 , wherein the (3R,4R,5S)-5-(difluoromethyl) piperidine-3,4-diol or salt thereof is administered about every 24 hours.